Shares of Ariad Pharmaceuticals Inc. gained 35.5 percent Friday after the European Committee for Medicinal Products for Human Use (CHMP) recommended that leukemia drug Iclusig (ponatinib) remain on the market in Europe.
It was a badly needed bit of good news for the beleaguered biotech, which has seen its stock hammered since early October. That’s when Ariad reported troubling safety data showing increased incidents of arterial blood clots in patients treated with Iclusig, resulting in a partial FDA clinical hold. The outlook worsened over the following weeks, as the company halted its Phase III EPIC study testing the pan-Bcr-Abl inhibitor in first-line chronic myelogenous leukemia (CML) – previusly hailed by management as the study that was supposed to have provided the risk-benefit data needed to alleviate the FDA’s concerns – and then, ultimately, suspended sales of Iclusig in the U.S. (See BioWorld Today, Oct. 10, 2013, Oct. 21, 2013, and Nov. 1, 2013.)
The stock (NASDAQ:ARIA), which had lost more than 80 percent of its value over the past month and a half, regained a little ground on the positive opinion out of Europe. Shares closed Friday at $3.78, up 99 cents.
In its positive recommendation following a review of Iclusig’s updated clinical data, the CHMP also suggested a number of recommendations for minimizing the risk of occlusive vascular events in patients taking the drug, including restricting the drug’s use in patients with a history of heart attack or stroke unless the potential benefits could outweigh the risks.
The CHMP also said patients’ cardiovascular status should be assessed and risk factors managed before starting treatment with Iclusig, and then continually monitored during treatment duration. In cases where patients’ hypertension cannot be controlled or evidence of vascular occlusion or thromboembolis occurs, treatment should be interrupted immediately.
J.P. Morgan analyst Cory Kasimov said the CHMP’s suggested measures “appear fairly unrestrictive and allow for some physician discretion. This is clearly a positive” for Ariad, he wrote in a research note, and “avoids a worst-case scenario.”
A final decision is expected to come from the European Commission within the next couple of months, but the commission typically rubberstamps CHMP recommendations.
Iclusig was approved in Europe in July for use in adults with chronic phase, accelerated phase or blast phase CML who are resistant to Sprycel (dasatinib, Bristol-Myers Squibb Co.) or Tasigna (nilotinib); who are intolerant to either Sprycel or Tasigna and for whom subsequent treatment with Gleevec (imatinib, Novartis AG) is not clinically appropriate; or who have the T3151 mutation. It’s also approved for use in patients with Philadelphia chromosome-positive acute lymphoblastic leukemia who are resistant or intolerant to Sprycel and for whom Gleevec is not approprirate, or who have the T3151 mutation.
In the U.S., investors will have to wait to find out Iclusig’s fate. Cambridge, Mass.-based Ariad said in recent third quarter earnings that it continues to work with the FDA.
The company, which cut 40 percent of its work force in the wake of Iclusig’s suspension in the U.S., expects to end the year with between $215 million and $220 million in cash.
In other CHMP decisions disclosed Friday:
• Astrazeneca plc, of London, and Bristol-Myers Squibb Co., of New York, said the CHMP adopted a positive opinion recommending approval of Xigduo (dapagliflozin and metformin hydrochloride) for adults with Type II diabetes mellitus as an adjunct to diet and exercise to improve glycemic control in patients inadequately controlled on current metformin-based treatment regimen or who are being treated with the combination of dapagliflozin and metformin as separate tablets.
• Biogen Idec Inc., of Weston, Mass., said the CHMP determined that the dimethyl fumarate in multiple sclerosis (MS) drug Tecfidera qualified as a new active substance, a designation that will provide 10 years of regulatory exclusivity in the European Union. “This removes a big overhand and we believe investors will now focus on Tecfidera [European Union] launch,” noted Deutsche Bank analyst Robyn Karnauskas in a research report. The CHMP previously recommended marketing authorization for Tecfidera as a first-line oral treatment for relapsing-remitting forms of MS. Shares of Biogen (NASDAQ:BIIB) jumped $33. 19, or 13.2 percent, to close Friday at $285.62. (See BioWorld Today, May 31, 2013.)
• Celgene International Sarl, of Boudry, Switzerland, a unit of Celgene Corp., said the CHMP adopted a positive opinion for Abraxane (nab-paclitaxel) in combination with gemcitabine for first-line treatment of adults with metastatic adenocarcinoma of the pancreas. The recommendation was based on the results of the Phase III MPACT study, which demonstrated a statistically significant improvement in median overall survival for the Abraxane/gemcitabine arm compared to gemcitabine alone and a 28 percent overall reduction in the risk of death. Abraxane gained approval in the U.S. for use in pancreatic cancer in September. (See BioWorld Today, Sept. 9, 2013.)
• Gilead Sciences Inc., of Foster City, Calif., said the CHMP adopted a positive opinion for Sovaldi (sofosbuvir), a once-daily oral nucleotide analogue polymerase inhibitor for the treatment of chronic hepatitis C virus infection in adults. In October, the drug was recommended for approval in the U.S., with a final decision expected by the FDA by Dec. 8. (See BioWorld Today, Oct. 28, 2013.)
• Merck KGgA, of Darmstadt, Germany, said its Merck Serono division reported that the CHMP issued a positive opinion on a variation to the Erbitux (cetuximab) product information, updating the assessment of benefit-risk in patients with metastatic colorectal cancer (mCRC). The committee recommended approval of the indication for Erbitux in the treatment of patients with RAS wild-type metastatic colorectal cancer based on the totality of data emerging on the role of mCRC RAS tumor status in the risk-benefit profile of the drug. The recommendation primarily refers to new biomarker data from the OPUS study.